2024 Summit

NEW CMT Summit Alert! Launch into CMT Clinical Trial Readiness with HNF!

by | Apr 1, 2024 | 0 comments

View Agenda Friday, June 7

​Breakfast

Keynote

Panel Discussion: CMT Biobank & Biomarkers for CMT Therapy Development

Patient Breakout Sessions & Feedback Sharing

Break

Panel Discussion: Innovating Digital Endpoints with Wearable Technology & Live Demo

CMT Lounges Open: In-Person Support, Resources & Socializing!

  • Surgical Consults – Dr. Glenn Pfeffer (by Appt.)
  • CMT Social – Meet & Greet w/ Vendor Tables
  • CMT Kids & Teens –  Meet & Greet w/ Resources
  • CMT Biobank – Donate Blood
  • CMT Genie – Genetic Testing Support
  • CMT GRIN – Registration & Survey Support

Lunch

Panel Discussion: Revolutionizing Clinical Trials with Video – If a Picture is Worth a Thousand Words, a Video is Worth a Million

Patient Video Breakout Session & Feedback Sharing

CMT Lounges Open: In-Person Support, Resources & Socializing!

  • Surgical Consults – Dr. Glenn Pfeffer (By appointment)
  • CMT Social – Meet & Greet w/ Vendor Tables
  • CMT Kids & Teens –  Meet & Greet w/Resources
  • CMT Biobank – Register & Donate Blood
  • CMT Genie – Genetic Testing Support
  • CMT GRIN – Registration & Survey Support

Presentation: New Techniques in Foot Corrective Surgeries for CMT, Glenn Pfeffer, MD

Workshop: Managing Mental Health & CMT

Panel Discussion: What’s blocking CMT Clinical Trials & Investments?

Closing Remarks, Q&A

View Agenda Saturday, June 8

Breakfast

Presentation: Turning Symptoms into Science

Interactive Breakout Session

Presentation: Repurposing Drugs for CMT

Patient Breakout Session

CMT Lounges Open: In-Person Support, Resources & Socializing!

  • Surgical Consults – Dr. Glenn Pfeffer (by Appt.)
  • CMT Social – Meet & Greet w/ Vendor Tables
  • CMT Kids & Teens –  Meet & Greet w/ Resources
  • CMT Biobank – Register & Donate Blood
  • CMT Genie – Genetic Testing Support
  • CMT GRIN – Registration & Survey Support

Lunch

Presentation: What Does it Take? A Fighter Mom’s Journey from Kitchen Table to Injection!

Presentation: What’s Next for Gene Therapies in CMT?

  • For Industry & Researchers Only: Surrogate Biomarkers
  • For Patients & Caregivers: What Does it Take to Participate in Future CMT Clinical Trials?

CMT Lounges Open: In-Person Support, Resources & Socializing!

  • Surgical Consults – Dr. Glenn Pfeffer (By appointment)
  • CMT Social – Meet & Greet w/ Vendor Tables
  • CMT Kids & Teens –  Meet & Greet w/ Resources
  • CMT Biobank – Register & Donate Blood
  • CMT Genie – Genetic Testing Support
  • CMT GRIN – Registration & Survey Support

Presentation: Overview of Upcoming Pipeline & Clinical Trials w/ Q&A

Closing Remarks

HNF Cocktail & Dinner Party

As NEW clinical trials emerge for CMT, the importance of optimizing measures, endpoints and the participant experience has never been more urgent! HNF is committed to derisking upcoming trials by bridging the gap between clinical trial study teams and patient perspectives and experiences.

This impact-driven summit is a chance for all patients, caregivers, clinicians, industry, and regulatory to connect with one another to exchange ideas, and engage in open discussions about the obstacles we face in designing and conducting CMT clinical trials. As HNF focuses on accelerating access to critical research data and specimens, this summit will also include on-site CMT Biobank collection.

Our aim is to create an all-in-one event for CMT Clinical Trial readiness with patients at the heart and center!

Session Topics include:

  • Patient and caregiver engagement
  • Patient experience
  • Designing a clinical trial protocol
  • Technology integration for clinical trials
  • Collaboration to share non-competitive patient insights

Onsite CMT Research activities:

HNF will maximize CMT data collection, including collecting biospecimen samples for our CMT Biobank and participating in HNF’s Global Registry for Inherited Neuropathies (GRIN).

What is the CMT Biobank?

HNF is excited to announce that we are continuing to collect CMT biospecimens to support therapy development. All CMT patient subtypes are welcome to participate!  There are still gaps in understanding the natural history of the disease correlation of genotype / phenotype, easy access to samples and data sharing to support translational research, and the validation of drug candidates and biomarkers for CMT.

**You will have a scheduled time during the summit for a phlebotomist to take your samples.

Learning Objectives:

  • Come together to share common ideas and open dialogue about the challenges we need to overcome so that we can work together and amplify the CMT patient voice and plan successful clinical trials.
  • Discover valuable insights into the patient’s experiences, challenges, and perspectives in navigating clinical trials, contributing to shaping future progress.
  • Bridge the gap between clinical trial study teams and patient perspectives and experiences.

#FacesofCMT will offer a unique opportunity to connect with patients or caregivers-some of whom have actively participated in clinical trials-in 1:1 meetings. Attendees can sign up on-site to meet with the participating patient advocates.

Learn more on this topic

Related Blog Posts

CMT Kids Video

The Hereditary Neuropathy Foundation is currently seeking 10 second video clips of CMT patients under the age of 16, performing daily activities.

Summit Panelist Spotlight: Mitchell Warner

Bracing is used in the management Charcot-Marie-Tooth to support and hold a part of the body compromised by muscle weakness, muscular atrophy, sensory impairment. Bracing is also used to help correct skeletal deformity.

Summit Panelist Spotlight: Brent Baker

With respect to CMT, Dr. Baker’s team has collaborated with Drs. Michael Sereda and Klaus Nave of the Max Planck Institute for Experimental Medicine (MPI) to utilize their CMT1A transgenic rat and established the initial CMT1A rat colony in the United States.

Join the conversation

Leave a Comment

0 Comments

Submit a Comment

Your email address will not be published. Required fields are marked *

Newsletter

Join for notifications on events, campaigns, & news